第4相治験のマスター

Abstract

Enhance Your Post-Marketing Trials:

Phase 4 is the fastest-growing area of clinical research today. At an annual growth rate of 23%, industry investment in post-marketing research is expected to top $12 billion in 2007. Rampant growth in Phase 4 research should come as no surprise, however. A changing regulatory environment, growing concerns about the safety of new medicines, and various uses for large-scale, real-world data on marketed drugs' safety and efficacy are primary drivers of the growth seen in the Phase 4 research environment today. Post-marketing research is an important element of commercialization that enables companies to expand existing markets, enter new markets, develop and deliver messaging that directly compares their products with the competition, and secure a niche position in crowded markets. What' s more, payer groups and regulators are both requiring more post-marketing data from drug companies.

Given the increasing importance of post-marketing research, continued growth expected for Phase 4 trials, and mounting competitive pressures, it is critical for companies to make full use of the information and data available to them. Phase IV Clinical Trials: Post-Marketing Study Management Structure, Strategy, and Benchmarks is a comprehensive post-marketing study management tool designed to provide industry executives with benchmark data and best practice information to inform key strategic and operational decisions in the areas of Phase 4:

• Trial management structure and oversight
• Research strategy
• Study design and execution
• Study timelines and cycle times
• Performance metrics and management
• Operational activities
• Trial budgeting and outsourcing
• Trial staffing

Table of Contents

Executive Summary

• Study Methodology and Definifions
• Profiled Companies and Therapeufic Areas
• Phase 4 Clinical Trials: Conclusions and Recommendations

Study Management Structure, Oversight and Strategy

• Phase 4 Study Management Structure
• Developing a Phase 4 Strategy

Phase 4 Trial timelines, Activities and Performance Metrics

• Post-Marketing Trial timelines
• Post-Marketing Trial Activities
• Performance Measurement

Budgets, Statfing and Outsourcing

• Phase 4 Study Budgets and Outsourcing
• Phase 4 Trial Stafing

CHARTS & GRAPHICS

Executive Summary

• Figure E.1: Percentage of Phase 4 Clinical Trial Budgets Outsourced, by Therapeutic Area
• Figure E.2: Percentage of Phase 4 Studies for Which Survey Respondents Submitted
• ASRs to FDA, 2005 and 2006

Study Management Sfructure. Oversight and Strategy

• Phase 4 Study Management Structure
  • Figure 1.1: Phase 4 Study Management Structure
  • Figure 1.2: Phase 4 Study Management Structure by Company Size
  • Figure 1.3: Company 7' s Phase 4 Management Structure
  • Figure 1.4: Company 12' s Phase 4 Management Structure
  • Figure 1.5: Total Number of Patients per Therapeutic Area
  • Figure 1.6: Average Number of Patients per Therapeutic Area Trial by Company Size
  • Figure 1.7: Number of Patients per Site
  • Figure 1.8: Average Number of Patients per Site by Company Size
  • Figure 1.9: Percentage of In-House Phase 4 Infrastructures
  • Figure 1.10: Percentage of In-House Phase 4 Infrastructures by Company Size
  • Figure 1.11: Percentage of Companies that Outsource Phase 4 Activities
  • Figure 1.12: Percentage of Companies that Outsource Phase 4 Activities by Company Size
• Developing a Phase 4 Strategy
  • Figure 1.13: Motivation for Conducting Phase 4 Studies
  • Figure 1.14: Average Number of Active Studies Per Motivation Category
  • Figure 1.15: Number of Active Phase 4 Studies by Company
  • Figure 1.16: Number of Active Phase 4 Studies Per Marketed Product by Company
  • Figure 1.17: Average Number of Active Phase 4 Studies Per Marketed Product by Company Size
  • Figure 1.18: Percentage Breakdown for Phase 4 Motivation by Company Size
  • Figure 1.19: Average Number of Active Phase 4 Trials by Motivation and Company Size
  • Figure 1.20: Number of Active Phase 4 Studies Per Small Company
  • Figure 1.21: Number of Active Phase 4 Studies Per Mid-Size Company
  • Figure 1.22: Number of Active Phase 4 Studies Per Large Company
  • Figure 1.23: Key Motivating Factors for Conducting Phase 4 Studies
  • Figure 1.24: Functional Oversight of Phase 4 Study Strategy
  • Figure 1.25: Functional Oversight of Phase 4 Study Strategy by Company Size
  • Figure 1.26: Participants' Self-Asse ssm e n ts of Study Pla nn ing and Design
  • Figure 1.27: Functional Oversight of Phase 4 Study Execution
  • Figure 1.28: Functional Oversight of Phase 4 Study Strategy by Company Size
  • Figure 1.29: Participants' Self-Assessments of Study Execution
  • Table 1.1: Participating Companies' Phase 4 Study Challenges

Phase 4 Trial Timelines, Activities and Performance Metrics

• Post-Marketing Trial Timelines
  • Figure 2.1: Percentage of NDAs with At Least One Post-Marketing Study C o m m itm e nt
  • Table 2.1: Post-Marketing Study Commitments (September 2005, FDA Federal Register)
  • Table 2.2: Post-Marketing Study Commitments from Fiscal Years 1990-2004
  • Figure 2.2: Products with Post-Marketing Studies Conducted
  • Figure 2.3: Products with Post-Marketing Studies Conducted by Company Size
  • Figure 2.4: Phase 4 Studies Initiated by All Surveyed Companies
  • Figure 2.5: Phase 4 Studies Initiated by Small Companies
  • Figure 2.6: Phase 4 Studies Initiated by Mid -Size Companies
  • Figure 2.7: Phase 4 Studies Initiated by Large Companies
  • Figure 2.8: Phase 4 Average & Projected Trial Duration
  • Figure 2.9: Percentage of Da ys Be yo nd Average Phase 4 Trial D ura tio n
  • Figure 2.10: Company Sponsored vs. Investigator Sponsored Post-Marketing US Studies
• Post-Marketing Trial Activities
  • Figure 2.11: Percentage s of Tim e Consumed by Trial Activities: Overall Average
  • Figure 2.12: Percentage s of Tim e Consumed by Trial Activities: Small Companies
  • Figure 2.13: Percentage s of Tim e Consumed by Trial Activities: Mid-Size Companies
  • Figure 2.14: Percentage s of Tim e Consumed by Trial Activities: Large Companies
  • Figure 2.15: Opportunities for Trial Acceleration : Overall Average
  • Figure 2.16: Opportunities for Trial Acceleration : Small Companies
  • Figure 2.17: Opportunities for Trial Acceleration : Mid-Size Companies
  • Figure 2.18: Opportunities for Trial Acceleration : Large Companies
  • Figure 2.19: Overall Performance Measurement Self-Ratings: All Activities
  • Figure 2.20: Performance Measurement Self-Ratings: Study Design & Concept Development
  • Figure 2.21: Performance Measurement Self-Ratings: Protocol Development/W riting
  • Figure 2.22: Performance Measurement Self-Ratings: Site Selection /Enrollment/Contracting
  • Figure 2.23: Performance Measurement Self-Ratings: Patient Enrollment
  • Figure 2.24: Performance Measurement Self-Ratings: CRO /Vendor Protocol
  • Figure 2.25: Performance Measurement Self-Ratings: CRO /Vendor Contracting
  • Figure 2.26: Performance Measurement Self-Ratings: Data Collection & Cleaning
  • Figure 2.27: Performance Measurement Self-Ratings: Statistical Analysis
• Performance Measurement
  • Figure 2.28: Self-Ratings: Measuring Performance
  • Figure 2.29: Self-Ratings: Using Performance Measurement to Effect Organizational Change
  • Figure 2.30: Self-Ratings: Measuring Performance (by Company Size )
  • Figure 2.31: Self-Ratings: Usin g Performance Measurement to Effect Organizational Change (by Company Size )
  • Figure 2.32: Percentage of Companies Tracking Efficiency/Operations Performance Metrics
  • Figure 2.33: Percentage of Companies Tracking Resource Metrics
  • Figure 2.34: Percentage of Companies Tracking Time Milestone Metrics
  • Table 2.3: Phase 4 Performance Metrics - Average Performance
  • Table 2.4: Phase 4 Performance Metrics - Target Performance
  • Table 2.5: Additional Phase 4 Performance Measures

Budgets, Staffing and Outosourcing

• Phase 4 Study Budgets and Outsourcing
  • Figure 3.1: Percentage of Total R& D Spending Allocated to Phase 4 Trials, by Company
  • Figure 3.2: Percentage of Total R& D Spending Allocated to Phase 4 Trials, by Company Size
  • Figure 3.3: Average Cost Per Patient of Phase 4 Clinical Trials in the US, by Therapeutic Area
  • Figure 3.4: Percentage of In-House Phase 4 Infrastructures
  • Figure 3.5: Percentage of In-House Phase 4 Infrastructures, by Company Size
  • Figure 3.6: Percentage of Phase 4 Clinical Trial Budgets Outsourced , by Therapeutic Area
• Phase 4 Trial Staffing
  • Figure 3.7: Average Numbers of Patients and Investigator Sites in Phase 4 Stafing Data
  • Figure 3.8: Average Patients-Per-Site and Patients-Per-CRA Ratios in Phase 4 Staffing Data
  • Table 3.1: Average Phase 4 Trial Stafing Across All Therapeutic Areas(FTEs)
  • Table 3.2: Phase 4 Trials (Trials 1-3)
  • Table 3.3: Phase 4 Trials: (Trials 4-6)
  • Table 3.4: Phase 4 Trials: (Trials 7-10)
  • Table 3.5: Phase 4 Trials: (Trials 11-14)
  • Table 3.6: Phase 4 Trials: (Trials 15-19)
  • Table 3.7: Phase 4 Trials: (Trials 20-23)
  • Table 3.8: Phase 4 Trials: (Trials 24-28)
  • Table 3.9: Phase 4 Trials: (Trials 29-31)
  • Table 3.10: Phase 4 Trials: (Trials 32-34)
  • Table 3.11: Phase 4 Trials: (Trials 35-39)
  • Figure 3.9: Average Patient Counts of Therapeutic Area-Specific Trials
  • Table 3.12: Average Phase 4 Trial Stafing by Therapeulic Area (FTEs)
  • Figure 3.10: Percentage of Staff Outsourced by Product Type
  • Table 3.13: Average Phase 4 Trial Stafing by Roduct Type (FTEs)
  • Figure 3.11: Average Patient Counts of Company Size-Specific Trials
  • Figure 3.12: Patients Per FTE by Company Size
  • Table 3.14: Average Phase 4 Trial Stafing by Company Size(FTEs)
  • Table 3.15: Average Phase 4 Trial Stafing by Trial Management Approach (FTEs)